'With that same spirit and focus on patient safety, we look forward to resuming the FORTIS clinical trial and the continued development of AT845 as an important potential new treatment for adults living with LOPD,' said
Following the clinical hold lift, Astellas is working on completing the clinical and regulatory activities necessary to resume dosing in the FORTIS clinical trial.
The clinical hold lift will have no impact on Astellas' financial forecasts of the current fiscal year ending
For more information, please see the press release 'Astellas Announces FDA Update on the FORTIS Clinical Trial of AT845 in Adults with Late-Onset Pompe Disease' issued on
About Pompe Disease
Pompe disease is a rare, severe, autosomal recessive metabolic disease characterized by progressive muscular degeneration. The overall incidence is estimated to be approximately 1 in 40,000 births1, although frequency and disease progression varies with age of onset, ethnicity and geography2. The disease is caused by mutations in the acid alpha-glucosidase (GAA) gene that prevent the production and function of a protein called acid alpha-glucosidase (GAA). GAA is responsible for metabolizing glycogen, and dysfunction or absence of this protein results in the accumulation of glycogen in tissues, primarily in the skeletal and cardiac muscles, where it causes damage to tissue structure and function. Currently, the only approved treatment for Pompe is enzyme replacement therapy (ERT), which is a chronic treatment delivered in bi-weekly infusions and relies solely on tissue uptake of GAA from plasma.
About AT845 for the treatment of Late-Onset Pompe Disease (LOPD)
Astellas is developing AT845, a novel gene replacement therapy using an AAV8 vector under a muscle-specific promotor to deliver a functional copy of the GAA gene, for the treatment of adult LOPD. AT845 is being investigated to determine whether it can deliver a functional GAA gene that is efficiently transduced to express GAA directly in tissues affected by the disease, including skeletal and cardiac muscle.
About FORTIS
FORTIS (NCT04174105) is a multicenter, open-label, ascending dose Phase I/II first-in-human clinical trial to determine if AT845 is safe and tolerable in adults with Late-Onset Pompe Disease (LOPD). The primary endpoints of the trial are safety and tolerability, as well as efficacy measures, including change in muscle GAA protein expression and enzyme activity from baseline. Secondary endpoints evaluate improvements in respiratory, endurance and quality of life measures.
About Astellas
About Astellas Gene Therapies
Astellas Gene Therapies is an
Astellas Cautionary Notes
In this press release, statements made with respect to current plans, estimates, strategies and beliefs and other statements that are not historical facts are forward-looking statements about the future performance of Astellas. These statements are based on management's current assumptions and beliefs in light of the information currently available to it and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in general economic conditions and in laws and regulations, relating to pharmaceutical markets, (ii) currency exchange rate fluctuations, (iii) delays in new product launches, (iv) the inability of Astellas to market existing and new products effectively, (v) the inability of Astellas to continue to effectively research and develop products accepted by customers in highly competitive markets, and (vi) infringements of Astellas' intellectual property rights by third parties. Information about pharmaceutical products (including products currently in development) which is included in this press release is not intended to constitute an advertisement or medical advice.
References
1. Kishnani, PS, et al. Pompe disease diagnosis and management guideline. Genetics in medicine: official journal of the
2. Ausems MG, et al. Frequency of glycogen storage disease type II in
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